New Treatments & Clinical Trials for Charcot-marie-tooth Disease

Charcot-Marie-Tooth disease is a group of inherited nerve disorders affecting about 1 in 2,500 people. It causes progressive weakness and numbness in the feet, legs, hands, and arms as the nerves that control muscles and carry sensation slowly degenerate. Until recently, no disease-modifying treatments existed—only physical therapy, braces, and surgery to manage symptoms.

What's actually going on in research

Trials are now testing gene therapy for specific CMT subtypes, antisense drugs that silence the mutated gene causing CMT1A, and small molecules that may protect nerve cells from damage. Researchers are also studying nerve regeneration approaches and testing drugs that cross from other rare diseases where similar nerve mechanisms are involved.

What to know before you search

Eligibility typically depends on confirmed genetic subtype, disease severity measured by strength and walking tests, age, and whether you've previously participated in gene therapy trials.

What types of trials are currently open

• Gene therapy trials — Testing one-time treatments that deliver a functional gene to replace the mutated one causing CMT in specific subtypes.
• Gene silencing trials — Testing drugs that reduce production of the abnormal protein in CMT1A, typically given as repeated injections under the skin.
• Neuroprotection trials — Testing pills or infusions that may slow nerve damage by supporting nerve cell health or reducing inflammation.
• Natural history studies — Following people with CMT over time to measure how the disease progresses and identify better outcome measures for future trials.
• Symptom management trials — Testing treatments for pain, muscle cramps, fatigue, and balance problems that affect daily life.